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Rett syndrome is an autism spectrum disorder caused by mosaicexpression of mutant copies of the X-linked MECP2 gene in neurons.However, neurons do not die, which suggests that this is nota neurodegenerative disorder. An important question for futuretherapeutic approaches to this and related disorders concernsphenotypic reversibility. Can viable but defective neurons berepaired, or is the damage done during development without normalMeCP2 irrevocable? Using a mouse model, we demonstrate robustphenotypic reversal, as activation of MeCP2 expression leadsto striking loss of advanced neurological symptoms in both immatureand mature adult animals.
1 Wellcome Trust Centre for Cell Biology, Edinburgh University, The King's Buildings, Edinburgh EH9 3JR, UK. 2 Neuroscience and Biomedical Systems, Institute of Biomedical and Life Sciences, West Medical Building, University of Glasgow, Glasgow G12 8QQ, UK.
* To whom correspondence should be addressed. E-mail: a.bird{at}ed.ac.uk
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